Drug intelligence / Profile preview

romiplostim + azacitidine

Development stage
Unknown
Lead developer
Amgen
Modality
Peptides, Small Molecules
Administration
Subcutaneous
01

Overview

**Romiplostim + azacitidine** is a pharmacological combination investigated for use in patients with myelodysplastic syndromes (MDS), particularly low- or intermediate-risk groups, to manage thrombocytopenia associated with azacitidine therapy. Romiplostim is a thrombopoietin receptor agonist (peptibody) that stimulates platelet production by binding to and activating the thrombopoietin receptor (c-Mpl). Azacitidine is a hypomethylating agent that inhibits DNA methylation, leading to the reactivation of tumor suppressor genes and cytotoxicity in abnormal hematopoietic cells. Clinical studies show that the combination can reduce the frequency of severe thrombocytopenia and platelet transfusion requirements during azacitidine therapy, with a generally tolerable safety profile. The combination is not currently approved but has been studied in phase 2 clinical trials for MDS.[1][2][3][4][5]

02

Targets

DNMT3A (DNA methyltransferase 3 alpha)TPOR (Thrombopoietin receptor)DNMT1 (DNA (cytosine-5)-methyltransferase 1)DNMT3B (DNA (cytosine-5)-methyltransferase 3B)

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